Induced pluripotent stem cells as a next-generation biomedical interface

Laboratory Investigation

Volumn 91, Issue 7, 2011, Pages 972-977

  Hankowski, Katherine E ^a   Hamazaki, Takashi ^a   Umezawa, Akihiro ^b   Terada, Naohiro ^a  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^b NATIONAL RESEARCH INSTITUTE FOR CHILD HEALTH AND DEVELOPMENT   (Japan)

Author keywords

clinical translational research; disease modeling; drug discovery; induced pluripotent stem cell; personalized medicine

Indexed keywords

BIOMEDICINE; CELL DIFFERENTIATION; CLINICAL RESEARCH; DISEASE MODEL; EMBRYONIC STEM CELL; GENETIC ASSOCIATION; HUMAN; IN VITRO STUDY; NONHUMAN; PHENOTYPE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; REVIEW; STEM CELL TRANSPLANTATION;

GENOME-WIDE ASSOCIATION STUDY; HUMANS; INDIVIDUALIZED MEDICINE; MODELS, BIOLOGICAL; MUTATION; PLURIPOTENT STEM CELLS; POLYMORPHISM, SINGLE NUCLEOTIDE;

EID: 79959770204     PISSN: 00236837     EISSN: 15300307     Source Type: Journal    
DOI: 10.1038/labinvest.2011.85     Document Type: Review

References (53)

1. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006;126:663-676. (Pubitemid 44233629)
2. Kiskinis E, Eggan K. Progress toward the clinical application of patientspecific pluripotent stem cells. J Clin Invest 2010;120:51-59.
3. Stadtfeld M, Hochedlinger K. Induced pluripotency: history, mechanisms, and applications. Genes Dev 2010;24:2239-2263.
4. Saha K, Jaenisch R. Technical challenges in using human induced pluripotent stem cells to model disease. Cell Stem Cell 2009;5:584-595.
5. Marchetto MC, Winner B, Gage FH. Pluripotent stem cells in neurodegenerative and neurodevelopmental diseases. Hum Mol Genet 2010;19:R71-R76.
6. Yoshida Y, Yamanaka S. Recent stem cell advances: induced pluripotent stem cells for disease modeling and stem cell-based regeneration. Circulation 2010;122:80-87.
7. Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007;131: 861-872. (Pubitemid 350138099)
8. Yu J, Vodyanik MA, Smuga-Otto K, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007;318: 1917-1920.
9. Park IH, Arora N, Huo H, et al. Disease-specific induced pluripotent stem cells. Cell 2008;134:877-886.
10. Dimos JT, Rodolfa KT, Niakan KK, et al. Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science 2008;321:1218-1221.
11. Ebert AD, Yu J, Rose Jr FF, et al. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2009;457:277-280.
12. Lee G, Papapetrou EP, Kim H, et al. Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. Nature 2009;461:402-406.
13. Marchetto MC, Carromeu C, Acab A, et al. A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells. Cell 2010;143:527-539.
14. Itzhaki I, Maizels L, Huber I, et al. Modelling the long QT syndrome with induced pluripotent stem cells. Nature 2011;471:225-229.
15. Moretti A, Bellin M, Welling A, et al. Patient-specific induced pluripotent stem-cell models for long-QT syndrome. N Engl J Med 2010;363:1397-1409.
16. Agarwal S, Loh YH, McLoughlin EM, et al. Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients. Nature 2010;464:292-296.
17. Ku S, Soragni E, Campau E, et al. Friedreich's ataxia induced pluripotent stem cells model intergenerational GAATTC triplet repeat instability. Cell Stem Cell 2010;7:631-637.
18. Okita K, Nakagawa M, Hyenjong H, et al. Generation of mouse induced pluripotent stem cells without viral vectors. Science 2008;322: 949-953.
19. Yu J, Hu K, Smuga-Otto K, et al. Human induced pluripotent stem cells free of vector and transgene sequences. Science 2009;324: 797-801.
20. Warren L, Manos PD, Ahfeldt T, et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 2010;7:618-630.
21. Zhou H, Wu S, Joo JY, et al. Generation of induced pluripotent stem cells using recombinant proteins. Cell Stem Cell 2009;4:381-384.
22. Lin T, Ambasudhan R, Yuan X, et al. A chemical platform for improved induction of human iPSCs. Nat Methods 2009;6:805-808.
23. Silva J, Barrandon O, Nichols J, et al. Promotion of reprogramming to ground state pluripotency by signal inhibition. PLoS Biol 2008;6:e253.
24. Thomson JA, Itskovitz-Eldor J, Shapiro SS, et al. Embryonic stem cell lines derived from human blastocysts. Science 1998;282:1145-1147. (Pubitemid 28516247)
25. Yu J, Thomson JA. Pluripotent stem cell lines. Genes Dev 2008;22:1987-1997.
26. Kim K, Doi A, Wen B, et al. Epigenetic memory in induced pluripotent stem cells. Nature 2010;467:285-290.
27. Stadtfeld M, Apostolou E, Akutsu H, et al. Aberrant silencing of imprinted genes on chromosome 12qF1 in mouse induced pluripotent stem cells. Nature 2010;465:175-181.
28. Polo JM, Liu S, Figueroa ME, et al. Cell type of origin influences the molecular and functional properties of mouse induced pluripotent stem cells. Nat Biotechnol 2010;28:848-855.
29. Murry CE, Keller G. Differentiation of embryonic stem cells to clinically relevant populations: lessons from embryonic development. Cell 2008;132:661-680. (Pubitemid 351260059)
30. Kobayashi T, Yamaguchi T, Hamanaka S, et al. Generation of rat pancreas in mouse by interspecific blastocyst injection of pluripotent stem cells. Cell 2010;142:787-799.
31. Nakagawa M, Koyanagi M, Tanabe K, et al. Generation of induced pluripotent stem cells without Myc from mouse and human fibroblasts. Nat Biotechnol 2008;26:101-106.
32. Soldner F, Hockemeyer D, Beard C, et al. Parkinson's disease patientderived induced pluripotent stem cells free of viral reprogramming factors. Cell 2009;136:964-977.
33. Hotta A, Cheung AY, Farra N, et al. Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency. Nat Methods 2009;6: 370-376.
34. Ye L, Chang JC, Lin C, et al. Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases. Proc Natl Acad Sci USA 2009;106:9826-9830.
35. Raya A, Rodriguez-Piza I, Guenechea G, et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 2009;460:53-59.
36. Maehr R, Chen S, Snitow M, et al. Generation of pluripotent stem cells from patients with type 1 diabetes. Proc Natl Acad Sci USA 2009;106:15768-15773.
37. Wang Y, Jiang Y, Liu S, et al. Generation of induced pluripotent stem cells from human beta-thalassemia fibroblast cells. Cell Res 2009;19:1120-1123.
38. Ye Z, Zhan H, Mali P, et al. Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders. Blood 2009;114:5473-5480.
39. Kazuki Y, Hiratsuka M, Takiguchi M, et al. Complete genetic correction of ips cells from Duchenne muscular dystrophy. Mol Ther 2010;18: 386-393.
40. Urbach A, Bar-Nur O, Daley GQ, et al. Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells. Cell Stem Cell 2010;6:407-411.
41. Carette JE, Pruszak J, Varadarajan M, et al. Generation of iPSCs from cultured human malignant cells. Blood 2010;115:4039-4042.
42. Carvajal-Vergara X, Sevilla A, D'Souza SL, et al. Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome. Nature 2010;465:808-812.
43. Rashid ST, Corbineau S, Hannan N, et al. Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells. J Clin Invest 2010;120:3127-3136.
44. Hargus G, Cooper O, Deleidi M, et al. Differentiated Parkinson patientderived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats. Proc Natl Acad Sci USA 2010;107:15921-15926.
45. Somers A, Jean JC, Sommer CA, et al. Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette. Stem Cells 2010; 28:1728-1740.
46. Chamberlain SJ, Chen PF, Ng KY, et al. Induced pluripotent stem cell models of the genomic imprinting disorders Angelman and Prader-Willi syndromes. Proc Natl Acad Sci USA 2010;107: 17668-17673.
47. Zhang N, An MC, Montoro D, et al. Characterization of human Huntington's disease cell model from induced pluripotent stem cells. PLoS Curr 2010;2:RRN1193.
48. Cooper O, Hargus G, Deleidi M, et al. Differentiation of human ES and Parkinson's disease iPS cells into ventral midbrain dopaminergic neurons requires a high activity form of SHH, FGF8a and specific regionalization by retinoic acid. Mol Cell Neurosci 2010;45: 258-266.
49. Yang J, Cai J, Zhang Y, et al. Induced pluripotent stem cells can be used to model the genomic imprinting disorder Prader-Willi syndrome. J Biol Chem 2010;285:40303-40311.
50. Zhang J, Lian Q, Zhu G, et al. A human iPSC model of Hutchinson Gilford Progeria reveals vascular smooth muscle and mesenchymal stem cell defects. Cell Stem Cell 2011;8:31-45.
51. Tolar J, Park IH, Xia L, et al. Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome). Blood 2011;117:839-847.
52. Tolar J, Xia L, Riddle MJ, et al. Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa. J Invest Dermatol 2011;131:848-856.
53. Pessach IM, Ordovas-Montanes J, Zhang SY, et al. Induced pluripotent stem cells: a novel frontier in the study of human primary immunodeficiencies. J Allergy Clin Immunol 2011; doi:10.1016/j.jaci.2010.11.008 (in press).